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eRAM

encyclopedia of Rare Disease Annotation for Precision Medicine




Disease muscular atrophy
Comorbidity C0026850|muscular dystrophy
Sentences 3
PubMedID- 25914643 In a mouse model of spinal muscular atrophy with a muscular dystrophy appearance of the skeletal muscle (hsa-cre, smnf7/f7 mice) receiving intravenous injection of a small number of afs cells were able to survive with drastic improvement of their muscle force (piccoli et al., 2012).
PubMedID- 22192627 In duchenne muscular dystrophy, the absence of dystrophin causes progressive muscle wasting and premature death.
PubMedID- 26280937 For duchenne muscular dystrophy, spinal muscular atrophy and myotonic dystrophy trials are ongoing to assess safety and efficacy, while in parallel preclinical studies are being conducted to identify ways to improve efficiency and delivery.

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